Seamless Clinical Trials with Doubly Adaptive Biased Coin Designs

Abstract

In addition to scientific questions, clinical trialists often explore or require other design features, such as increasing the power while controlling the type I error rate, minimizing unnecessary exposure to inferior treatments, and comparing multiple treatments in one clinical trial. We propose implementing adaptive seamless design (ASD) with response adaptive randomization (RAR) to satisfy various clinical trials’ design objectives. However, the combination of ASD and RAR poses a challenge in controlling the type I error rate. In this paper, we investigated how to utilize the advantages of the two adaptive methods and control the type I error rate. We offered the theoretical foundation for this procedure. Numerical studies demonstrated that our methods could achieve efficient and ethical objectives while controlling the type I error rate.

Editorial. Design and Analysis of Experiments for Data Science

“…Clinical trialists often grapple with the need to balance statistical power, ethical considerations, and control over the type I error rate. Zhu et al [11] propose an adaptive seamless design (ASD) in conjunction with response adaptive randomization (RAR) to address these challenges. This innovative approach demonstrates how it is possible to achieve efficient and ethical objectives while maintaining control over the type I error rate, a crucial aspect in clinical trials.…”